Liver Transplantation in Children With Propionic Acidemia: Medium-Term Outcomes

被引:24
作者
Curnock, Richard [1 ]
Heaton, Nigel D. [2 ]
Vilca-Melendez, Hector [2 ]
Dhawan, Anil [3 ]
Hadzic, Nedim [3 ]
Vara, Roshni [1 ,3 ]
机构
[1] Evelina Childrens Hosp, Paediat Inherited Metab Dis, London, England
[2] Kings Coll Hosp London, Inst Liver Studies, Liver Transplantat Surg, London, England
[3] Kings Coll Hosp London, Paediat Liver Gastroenterol & Nutr Ctr, London, England
关键词
MANAGEMENT; PATIENT; COMPLICATIONS; SURVIVAL;
D O I
10.1002/lt.25679
中图分类号
R57 [消化系及腹部疾病];
学科分类号
摘要
Liver transplantation (LT) for patients with propionic acidemia (PA) is an emerging therapeutic option. We present a retrospective review of patients with PA who underwent LT at a tertiary liver center between 1995 and 2015. A total of 14 children were identified (8 males) with median age at initial presentation of 3 days (range, 0-77 days). Pretransplant median protein restriction was 1 g/kg/day (range, 0.63-1.75 g/kg/day), 71% required supportive feeding, and 86% had developmental delay. Frequent metabolic decompensations (MDs) were the main indication for LT with a median age at transplantation of 2.4 years (range, 0.8-7.1 years). Only 1 graft was from a living donor, and 13 were from deceased donors (4 auxiliary). The 2-year patient survival was 86%, and overall study and graft survival was 79% and 69%, respectively. Three patients died after LT: at 43 days (biliary peritonitis), 225 days (acute-on-chronic rejection with multiorgan failure), and 13.5 years (posttransplant lymphoproliferative disease). Plasma glycine and propionylcarnitine remained elevated but reduced after transplant. Of 11 survivors, 5 had at least 1 episode of acute cellular rejection, 2 sustained a metabolic stroke (with full recovery), and 3 developed mild cardiomyopathy after LT. All have liberalized protein intake, and 9 had no further MDs: median episodes before transplant, 4 (range, 1-30); and median episodes after transplant, 0 (range, 0-5). All survivors made some developmental progress after LT, and none worsened at a median follow-up of 5.8 years (range, 2-23 years). LT in PA significantly reduces the frequency of MDs, can liberalize protein intake and improve quality of life, and should continue to be considered in selected cases.
引用
收藏
页码:419 / 430
页数:12
相关论文
共 38 条
[1]   Left ventricular assist device as bridge to liver transplantation in a patient with propionic acidemia and cardiogenic shock [J].
Ameloot, Koen ;
Vlasselaers, Dirk ;
Dupont, Matthias ;
Meersseman, Wouter ;
Desmet, Lars ;
Vanhaecke, Johan ;
Vermeer, Nina ;
Meyns, Bart ;
Pirenne, Jacques ;
Cassiman, David ;
De Laet, Corinne ;
Goyens, Philippe ;
Malekzadeh-Milan, Sophie G. ;
Biarent, Dominique ;
Meulemans, Ann ;
Debray, Francois-Guillaume .
JOURNAL OF PEDIATRICS, 2011, 158 (05) :866-867
[2]   Reversal of cardiomyopathy in propionic acidemia after liver transplantation: a 10-year follow-up [J].
Arrizza, Chiara ;
De Gottardi, Andrea ;
Foglia, Ezio ;
Baumgartner, Matthias ;
Gautschi, Matthias ;
Nuoffer, Jean-Marc .
TRANSPLANT INTERNATIONAL, 2015, 28 (12) :1447-1450
[3]   Evaluation and management of patients with propionic acidemia undergoing liver transplantation: A comprehensive review [J].
Barshes, Neal R. ;
Vanatta, Jason M. ;
Patel, Akash J. ;
Carter, Beth A. ;
O'Mahony, Christine A. ;
Karpen, Saul J. ;
Goss, John A. .
PEDIATRIC TRANSPLANTATION, 2006, 10 (07) :773-781
[4]   Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia [J].
Baumgartner, Matthias R. ;
Hoerster, Friederike ;
Dionisi-Vici, Carlo ;
Haliloglu, Goknur ;
Karall, Daniela ;
Chapman, Kimberly A. ;
Huemer, Martina ;
Hochuli, Michel ;
Assoun, Murielle ;
Ballhausen, Diana ;
Burlina, Alberto ;
Fowler, Brian ;
Gruenert, Sarah C. ;
Gruenewald, Stephanie ;
Honzik, Tomas ;
Merinero, Begona ;
Perez-Cerda, Celia ;
Scholl-Buergi, Sabine ;
Skovby, Flemming ;
Wijburg, Frits ;
MacDonald, Anita ;
Martinelli, Diego ;
Sass, Joern Oliver ;
Valayannopoulos, Vassili ;
Chakrapani, Anupam .
ORPHANET JOURNAL OF RARE DISEASES, 2014, 9
[5]   Early and Late Complications After Liver Transplantation for Propionic Acidemia in Children: A Two Centers Study [J].
Charbit-Henrion, F. ;
Lacaille, F. ;
McKiernan, P. ;
Girard, M. ;
de Lonlay, P. ;
Valayannopoulos, V. ;
Ottolenghi, C. ;
Chakrapani, A. ;
Preece, M. ;
Sharif, K. ;
Chardot, C. ;
Hubert, P. ;
Dupic, L. .
AMERICAN JOURNAL OF TRANSPLANTATION, 2015, 15 (03) :786-791
[6]   Liver Transplantation for Propionic Acidemia and Methylmalonic Acidemia: Perioperative Management and Clinical Outcomes [J].
Critelli, Kristen ;
McKiernan, Patrick ;
Vockley, Jerry ;
Mazariegos, George ;
Squires, Robert H. ;
Soltys, Kyle ;
Squires, James E. .
LIVER TRANSPLANTATION, 2018, 24 (09) :1260-1270
[7]   Autism spectrum disorders in propionic acidemia patients [J].
de la Batie, Caroline Dejean ;
Barbier, Valerie ;
Roda, Celina ;
Brassier, Anais ;
Arnoux, Jean-Baptiste ;
Valayannopoulos, Vassili ;
Guemann, Anne-Sophie ;
Pontoizeau, Clement ;
Gobin, Stephanie ;
Habarou, Florence ;
Lacaille, Florence ;
Bonnefont, Jean-Paul ;
Canoui, Pierre ;
Ottolenghi, Chris ;
De Lonlay, Pascale ;
Ouss, Lisa .
JOURNAL OF INHERITED METABOLIC DISEASE, 2018, 41 (04) :623-629
[8]   Long-term outcome of liver retransplantation in children [J].
Deshpande, PR ;
Rela, M ;
Girlanda, R ;
Bowles, MJ ;
Muiesan, P ;
Dhawan, A ;
Mieli-Vergani, G ;
Heaton, ND .
TRANSPLANTATION, 2002, 74 (08) :1124-1130
[9]   Propionic acidemia: neonatal versus selective metabolic screening [J].
Gruenert, S. C. ;
Muellerleile, S. ;
de Silva, L. ;
Barth, M. ;
Walter, M. ;
Walter, K. ;
Meissner, T. ;
Lindner, M. ;
Ensenauer, R. ;
Santer, R. ;
Bodamer, O. A. ;
Baumgartner, M. R. ;
Brunner-Krainz, M. ;
Karall, D. ;
Haase, C. ;
Knerr, I. ;
Marquardt, T. ;
Hennermann, J. B. ;
Steinfeld, R. ;
Beblo, S. ;
Koch, H. G. ;
Konstantopoulou, V. ;
Scholl-Buergi, S. ;
van Teeffelen-Heithoff, A. ;
Suormala, T. ;
Sperl, W. ;
Kraus, J. P. ;
Superti-Furga, A. ;
Schwab, K. O. ;
Sass, J. O. .
JOURNAL OF INHERITED METABOLIC DISEASE, 2012, 35 (01) :41-49
[10]   Propionic acidemia: clinical course and outcome in 55 pediatric and adolescent patients [J].
Gruenert, Sarah C. ;
Muellerleile, Stephanie ;
De Silva, Linda ;
Barth, Michael ;
Walter, Melanie ;
Walter, Kerstin ;
Meissner, Thomas ;
Lindner, Martin ;
Ensenauer, Regina ;
Santer, Rene ;
Bodamer, Olaf A. ;
Baumgartner, Matthias R. ;
Brunner-Krainz, Michaela ;
Karall, Daniela ;
Haase, Claudia ;
Knerr, Ina ;
Marquardt, Thorsten ;
Hennermann, Julia B. ;
Steinfeld, Robert ;
Beblo, Skadi ;
Koch, Hans-Georg ;
Konstantopoulou, Vassiliki ;
Scholl-Buergi, Sabine ;
van Teeffelen-Heithoff, Agnes ;
Suormala, Terttu ;
Sperl, Wolfgang ;
Kraus, Jan P. ;
Superti-Furga, Andrea ;
Schwab, Karl Otfried ;
Sass, Joern Oliver .
ORPHANET JOURNAL OF RARE DISEASES, 2013, 8